학술논문
Luspatercept for the treatment of anaemia in non-transfusion-dependent β-thalassaemia (BEYOND): a phase 2, randomised, double-blind, multicentre, placebo-controlled trial
Document Type
Article
Author
Taher, Ali T; Cappellini, Maria Domenica; Kattamis, Antonis; Voskaridou, Ersi; Perrotta, Silverio; Piga, Antonio G; Filosa, Aldo; Porter, John B; Coates, Thomas D; Forni, Gian Luca; Thompson, Alexis A; Tartaglione, Immacolata; Musallam, Khaled M; Backstrom, Jay T; Esposito, Oriana; Giuseppi, Ana Carolina; Kuo, Wen-Ling; Miteva, Dimana; Lord-Bessen, Jennifer; Yucel, Aylin; Zinger, Tatiana; Shetty, Jeevan K; Viprakasit, Vip; Buaboonnam, Jassada; Ekwattanakit, Supachai; Khunhapinant, Archrob; Loka, Efthalia; Moraki, Maria; Flevari, Pagona; Dimopoulou, Maria; Bartzi, Vasiliki; Daadaa, Hisham; El Hasbani, Georges; Koussa, Suzanne; Tartaglione, Immacolata; Ammendola, Federica; Scianguetta, Saverio; Puglia, Marta; Ferrara, Ilaria; Ferrero, Giovanni; Gaglioti, Carmen; Longo, Filomena; Turrini, Silvia; Voi, Vincenzo; Cassinerio, Elena; De, Anna; Graziadei, Giovanna; Marcon, Alessia; Migone De Amicis, Margherita; Motta, Irene; Cinque, Patrizia; Pannone, Bruno; Ricchi, Paolo; Balocco, Manuela; Carrara, Paola; Della Rovere, Francesco; Lamagna, Martina; Pinto, Valeria; Quintino, Sabrina; Eleftheriou, Perla; Garbowski, Maciej; de Kreuk, Arne; Carson, Susan; Denton, Christopher; Hofstra, Tom; Veluswamy, Sayany; Wood, John; Badawy, Sherif; Bercovitz, Rachel; Bhat, Rukhmi; Calamaras, Diane; Liem, Robert; Mack, Astrid
Source
The Lancet Haematology; October 2022, Vol. 9 Issue: 10 pe733-e744, 12p
Subject
Language
ISSN
23523026
Abstract
In patients with non-transfusion-dependent β-thalassaemia, haemoglobin concentrations lower than 10 g/dL are associated with a higher risk of morbidity, mortality, and impaired quality of life. No drugs are specifically approved for anaemia management in patients with non-transfusion-dependent β-thalassaemia, other than transfusion therapy administered infrequently in accordance with patients’ needs. We assessed the efficacy and safety of luspatercept versus placebo in patients with non-transfusion-dependent β-thalassaemia.